The Ministry of Health and Welfare said on the 21st that it held the 11th Advanced Regenerative Medicine and Advanced Biopharmaceuticals Review Committee for 2025 on the 20th and approved three of the eight clinical studies submitted by regenerative medicine institutions as "appropriate." The ministry determined that, of the remaining five, three were "inappropriate" and two required further discussion.
The studies deemed appropriate this time are three in total: ▲ treatment with allogeneic umbilical cord-derived mesenchymal stem cells for patients with partial-thickness rotator cuff tears ▲ administration of induced pluripotent stem cell (iPSC)-based chondrocyte aggregates (MIUChon) for patients with knee osteoarthritis ▲ administration of CD19 CAR-T for pediatric and adolescent patients with intractable systemic lupus erythematosus (SLE).
The first study is a high-risk clinical study on treatment with allogeneic umbilical cord-derived mesenchymal stem cells administered by injection without surgery to patients with partial-thickness rotator cuff tears. The safety of these cells has already been confirmed in a prior study (R-3-0013) involving patients with large tears. This study will evaluate efficacy by setting up a control group and using a randomized, double-blind design.
The second study involves injecting induced pluripotent stem cell (iPSC)-derived chondrocyte aggregates (MIUChon) into the knee joint cavity. It is a follow-up study conducted after confirming short-term safety in a small-scale prior clinical study (R-3-0012), and it expands the number of participants and applies a randomized, double-blind, placebo-controlled design to meet global safety and statistical verification standards.
The third study is a clinical trial administering the immune cell therapy "CD19 CAR-T" to pediatric and adolescent patients with intractable systemic lupus erythematosus (SLE).
CAR-T therapy modifies a patient's immune cells to make them directly attack cancer cells. Lupus is a rare, intractable disease in which the immune system attacks the body's organs.
CD19 CAR-T cells are produced by introducing a gene designed to recognize the B-cell surface antigen "CD19" into a patient's immune T cells. They work by selectively locating and attacking malignant B cells.
Conventional CAR-T therapy has mainly been applied to neoplastic diseases, but clinical trials are now underway globally for adult, pediatric, and adolescent patients with lupus.
In Korea, a CAR-T clinical trial for adult lupus patients was first approved in Aug., and this is the first study in which children and adolescents can participate. Because pediatric- and adolescent-onset lupus is more severe than in adults and has a higher rate of major organ involvement, the approval is seen as significant.
The Ministry of Food and Drug Safety applied an expedited and combined review process to all three studies, as they fall under high-risk clinical studies, and notified the committee that the submitted data were reasonable. Regenerative medicine institutions will begin the clinical studies after receiving the ministry's approval notice.
Kim U-gi, secretary general of the Advanced Regenerative Medicine Review Committee, said, "We reviewed studies on a range of serious diseases from cancer to musculoskeletal and rare and intractable diseases," and added, "We are making comprehensive judgments on safety, efficacy, and ethics to provide patients with safer access to advanced regenerative medicine." Kim also said, "We are operating pre-consultations to help establish research and treatment plans, so please make active use of them."